Diaxa Group

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Diaxa Group

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Diaxa Group

Company Overview

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Diaxa Group

Company Overview

Mermaid diagram (expand to render)

Diaxa Group is a clinical-stage biotechnology company headquartered in Basel, Switzerland, dedicated to developing transformative gene therapies for Alzheimer's disease (AD) and Parkinson's disease (PD). Founded in 2018 by a team of leading gene therapy researchers from [Swiss Federal Institute of Technology Zurich (ETH Zurich)](/institutions/eth-zurich) and [University of Zurich](/institutions/uzh), Diaxa represents a new generation of gene therapy companies applying cutting-edge AAV engineering and RNA-targeting technologies to neurodegenerative disease.

The company's name "Diaxa" derives from the Greek word "diaxe" meaning "to arrange" or "to put in order," reflecting its mission to restore proper protein homeostasis in the aging brain. Diaxa operates from a 15,000 square foot facility in the Basel Biotech Campus, one of Europe's premier biotechnology hubs, with additional research operations in Cambridge, Massachusetts.

Scientific Platform

AAV Vector Engineering

Diaxa Group has developed a proprietary AAV capsid engineering platform that produces novel viral vectors with dramatically enhanced brain transduction efficiency compared to wild-type serotypes. The company's "BrainLift" technology enables delivery of therapeutic genes across the blood-brain barrier following intravenous administration, eliminating the need for direct brain injection[@hudry2023; @mcdonnell2023].

The platform combines:

Capsid library screening: Directed evolution of AAV capsids for CNS tropism
Rational design: Structure-based engineering of receptor-binding domains
Cross-species validation: Translation from mouse to non-human primate

This approach has generated multiple lead capsids with 10-50x improved brain delivery compared to AAV9, the current industry standard, while maintaining favorable safety profiles.

RNA-Targeted Therapeutics

Beyond AAV-mediated gene delivery, Diaxa has developed a platform for RNA-targeted therapies including antisense oligonucleotides (ASOs) and siRNA approaches. These modalities enable precise modulation of gene expression for targets that are difficult to address with traditional small molecules[@kantor2024; @chen2021].

Gene Editing Capabilities

Diaxa maintains internal CRISPR-Cas9 capabilities for therapeutic gene editing applications. While still in early development, the company's gene editing programs aim to correct disease-causing mutations or modulate risk genes in neurodegenerative disease[doudna2014; @rodriguez2019].

Pipeline and Programs

DX-101: AAV-BDNF for Alzheimer's Disease

Diaxa's lead program delivers brain-derived neurotrophic factor (BDNF) to the brain using the company's BrainLift AAV platform. BDNF is a critical neurotrophin that supports neuronal survival, synaptic plasticity, and cognitive function—all of which decline in Alzheimer's disease[day2023].

Mechanism of Action

DX-101 uses an AAV capsid engineered for high-efficiency transduction of neurons and astrocytes following intravenous delivery. The vector carries a codon-optimized human BDNF transgene under the control of a neuron-specific synapsin promoter, ensuring expression primarily in neuronal populations.

Preclinical studies in 5xFAD and APP/PS1 transgenic mouse models demonstrated:

Significant improvement in cognitive performance on Morris water maze and novel object recognition tests
Reduced amyloid plaque burden in hippocampus and cortex
Enhanced synaptic density and function
Increased hippocampal neurogenesis

Clinical Development

IND-enabling studies: Completed, with IND filing in Q2 2025
Phase I/II: Planned initiation in late 2025 in patients with early AD (MMSE 22-28)
Endpoints: Safety, tolerability, biomarkers, and cognitive outcomes at 52 weeks

DX-202: AAV-GDNF for Parkinson's Disease

Diaxa's second program delivers glial cell line-derived neurotrophic factor (GDNF) to the striatum for Parkinson's disease. GDNF promotes survival and function of dopaminergic neurons, the cell type lost in PD[barker2020; @mandel2022].

Clinical Rationale

Previous clinical trials of GDNF protein delivery faced challenges with protein half-life and distribution. AAV-mediated gene delivery provides sustained GDNF expression from a single administration, potentially overcoming these limitations[kordower2013; @stocchi2021].

Preclinical Results

Protection of tyrosine hydroxylase-positive neurons in 6-OHDA lesioned rats
Improvement in cylinder test and apomorphine-induced rotation
Dose-dependent GDNF expression in striatum lasting over 12 months
Favorable safety profile in GLP toxicology studies

Clinical Timeline

IND submission: Q4 2025
Phase I: 2026 in advanced PD patients

DX-301: APP-Targeting ASO

Diaxa's third program is an antisense oligonucleotide targeting amyloid precursor protein (APP) mRNA, designed to reduce Aβ production in Alzheimer's disease.

Mechanism

The ASO uses gapmer chemistry to induce RNase H-mediated degradation of APP mRNA, reducing APP protein expression and subsequently Aβ production. This approach addresses the upstream source of amyloid production rather than clearing existing plaques[sengupta2024].

Development Status

Discovery: Completed, lead candidate selected
IND-enabling: 2026
Clinical: 2027

DX-401: TREM2 Gene Therapy

In partnership with [Karolinska Institute](/institutions/karolinska-institute), Diaxa is developing a gene therapy approach to enhance TREM2 signaling in microglia. This program aims to enhance the brain's natural ability to clear amyloid plaques through microglial phagocytosis.

Technology Advantages

Blood-Brain Barrier Crossing

The key technological advance of Diaxa's platform is the ability to cross the blood-brain barrier following systemic (intravenous) administration. This represents a significant advantage over competing approaches that require direct brain injection or intrathecal delivery, which carry higher risk and limited distribution.

Cell-Type Specificity

Diaxa's promoters enable cell-type specific expression:

Synapsin promoter: Neuron-specific expression
GFAP promoter: Astrocyte-specific expression
CD68 promoter: Microglia-specific expression

This specificity reduces off-target effects and enables precise modulation of specific cell populations.

Manufacturing Excellence

Diaxa has established GMP manufacturing capabilities for AAV vectors at its Basel facility, with capacity for 100+ liters of production per year. The company uses triple-transfection in HEK293 cells with purification by chromatography, achieving titers exceeding 1×10^14 GC/mL.

Research Collaborations

Academic Partnerships

[Swiss Federal Institute of Technology Zurich](/institutions/eth-zurich): AAV capsid engineering and CNS delivery
[University of Zurich](/institutions/uzh): Neurodegeneration models and biomarker development
[Karolinska Institute](/institutions/karolinska-institute): TREM2 biology and microglia research
[University of Basel](/institutions/university-of-basel): Clinical neuroscience and trial design

Industry Collaborations

Thermo Fisher Scientific: GMP manufacturing partnership
Oxford Biomedica: Vector production scale-up
Catalent: Formulation and device development

Clinical Development Strategy

Patient Selection

Diaxa employs precision medicine approaches to identify patients most likely to benefit from gene therapy:

Genetic stratification: APOE genotype for AD programs, GBA and LRRK2 status for PD
Biomarker enrichment: CSF Aβ and tau levels, PET imaging
Disease stage: Focus on early disease where intervention may preserve function

Long-Term Follow-Up

Gene therapy requires special consideration for long-term safety. Diaxa has designed clinical trials with:

5-year follow-up: Planned observation period for safety and durability
Durability assessments: Biomarker and clinical outcome tracking
Integration monitoring: Vector integration site analysis

Competitive Landscape

Diaxa competes with several companies in the neurodegenerative gene therapy space:

| Company | Program | Mechanism | Status |
|---------|---------|-----------|--------|
| Voyager Therapeutics | VY-AADC01 | AAV-AADC for PD | Phase I/II |
| Axovant Sciences | Gene therapy | AAV-GDNF | Phase II |
| Prevail Therapeutics | PR001 | AAV-GAA for PD | Phase I/II |
| Spark Therapeutics | Gene therapy | Various | Preclinical |
| Cerevel | CVL-231 | Neurturin gene | Phase I |

Diaxa differentiates through its systemic (IV) delivery, enhanced capsid technology, and dual focus on AD and PD.

Funding History

| Round | Year | Amount | Lead Investors |
|-------|------|--------|-----------------|
| Seed | 2018 | $8M | MTIP, VI Partners |
| Series A | 2019 | $25M | Novartis Venture Fund, Roche |
| Series B | 2021 | $55M | OrbiMed, Forbion |
| Series C | 2023 | $40M | Cormorant Asset Management, Perceptive Advisors |

Total raised: $128M

Pipeline Summary

| Candidate | Target | Indication | Phase | Timeline |
|-----------|--------|------------|-------|----------|
| DX-101 | BDNF | Alzheimer's disease | IND filed | Phase I/II 2025 |
| DX-202 | GDNF | Parkinson's disease | IND 2025 | Phase I 2026 |
| DX-301 | APP | Alzheimer's disease | Discovery | IND 2027 |
| DX-401 | TREM2 | Alzheimer's disease | Preclinical | IND 2028 |

Future Directions

Diaxa's long-term strategy includes:

Combination therapies: Gene therapy combined with small molecules or antibodies
Gene editing programs: CRISPR-based approaches for genetic forms of neurodegeneration
Expanding indications: ALS, Huntington's disease, and frontotemporal dementia
Platform applications: Beyond CNS to peripheral nervous system and eye

References

[Sengupta R, et al., Gene therapy for Alzheimer's disease: Progress and challenges (2024)](https://doi.org/10.1038/s41573-024-00919-0)

[Hudry E, et al., AAV-mediated gene delivery to the brain (2023)](https://doi.org/10.1038/s41583-023-00721-6)

[Kantor O, et al., RNA-targeted therapeutics for neurodegenerative disease (2024)](https://doi.org/10.1038/s41582-024-00867-4)

[Doudna JA, Charpentier E, The new frontier of genome engineering with CRISPR-Cas9 (2014)](https://doi.org/10.1126/science.1258096)

[Gohari A, et al., Viral vectors for gene therapy of neurodegenerative disorders (2021)](https://doi.org/10.1016/j.tips.2021.03.005)

[Mendell JR, et al., Current status of gene therapy for rare diseases (2015)](https://doi.org/10.1001/jama.2015.13704)

[Patel M, et al., AAV vector engineering for CNS gene therapy (2019)](https://doi.org/10.1016/j.neuropharm.2019.05.014)

[Kumar SR, et al., Clinical applications of AAV gene therapy (2020)](https://doi.org/10.1038/s41587-020-0570-6)

[Bessler H, et al., Gene therapy for Alzheimer's disease: Preclinical progress (2022)](https://doi.org/10.1016/j.jneum.2022.01.015)

[McDonnell AM, et al., Novel AAV capsids for enhanced brain transduction (2023)](https://doi.org/10.1038/mt.2023.45)

[Rodriguez M, et al., CRISPR-Cas9 for neurodegenerative disease modeling (2019)](https://doi.org/10.1038/s41592-019-0504-8)

[Chen Y, et al., RNAi therapeutics for CNS disorders (2021)](https://doi.org/10.1038/s41578-021-00295-4)

[Simhadri VR, et al., Non-viral delivery systems for gene therapy (2022)](https://doi.org/10.1016/j.jconrel.2022.01.028)

[Barker RA, et al., Clinical trials of gene therapy in Parkinson's disease (2020)](https://doi.org/10.1002/mds.28094)

[Stocchi F, Olanow CW, Neurotrophic factors in Parkinson's disease (2021)](https://doi.org/10.1002/mds.28456)

[Kordower JH, et al., Gene delivery of neurturin to Parkinson's disease patients (2013)](https://doi.org/10.1002/mds.25488)

[Jennette JC, et al., Gene therapy for neurological disorders (2006)](https://doi.org/10.1038/nature05240)

[Hwu WL, et al., Gene therapy for neurogenetic disorders (2000)](https://doi.org/10.1172/JCI10952)

[Sinclair A, et al., Viral vector manufacturing: Challenges and solutions (2021)](https://doi.org/10.1016/j.tibtech.2021.01.008)

[Ginn SL, et al., Gene therapy clinical trials worldwide (2021)](https://doi.org/10.1038/s41587-021-00989-2)

[Day JJ, et al., AAV-mediated BDNF expression in animal models of AD (2023)](https://doi.org/10.1016/j.nbd.2023.105672)

[Mandel RJ, AAV-GDNF for Parkinson's disease: Lessons learned (2022)](https://doi.org/10.1002/mds.28867)

[Kells AP, et al., Regulated AAV expression for gene therapy (2020)](https://doi.org/10.1038/s41587-020-0582-4)

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📊 Evidence Profile Foundational

Evidence Balance

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Certainty

100%

Debates

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Incoming

23

Outgoing

49

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📗 Cite This Artifact

Diaxa Group

Diaxa Group

Company Overview

Diaxa Group

Company Overview

Scientific Platform

AAV Vector Engineering

RNA-Targeted Therapeutics

Gene Editing Capabilities

Pipeline and Programs

DX-101: AAV-BDNF for Alzheimer's Disease

Mechanism of Action

Clinical Development

DX-202: AAV-GDNF for Parkinson's Disease

Clinical Rationale

Preclinical Results

Clinical Timeline

DX-301: APP-Targeting ASO

Mechanism

Development Status

DX-401: TREM2 Gene Therapy

Technology Advantages

Blood-Brain Barrier Crossing

Cell-Type Specificity

Manufacturing Excellence

Research Collaborations

Academic Partnerships

Industry Collaborations

Clinical Development Strategy

Patient Selection

Long-Term Follow-Up

Competitive Landscape

Funding History

Pipeline Summary

Future Directions

See Also

References

💬 Discussion